JOP20210276A1 - أنظمة تحرير جيني لتعديل جين scn9a أو scn10a وطرق لاستخدامها - Google Patents

أنظمة تحرير جيني لتعديل جين scn9a أو scn10a وطرق لاستخدامها

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Publication number
JOP20210276A1
JOP20210276A1 JOP/2021/0276A JOP20210276A JOP20210276A1 JO P20210276 A1 JOP20210276 A1 JO P20210276A1 JO P20210276 A JOP20210276 A JO P20210276A JO P20210276 A1 JOP20210276 A1 JO P20210276A1
Authority
JO
Jordan
Prior art keywords
gene
editing systems
scn9a
scn10a
modifying
Prior art date
Application number
JOP/2021/0276A
Other languages
English (en)
Inventor
Sophia Espanola
Sheena Saayman
Sarah Jean Spencer
Stuart Cahalan
Angela Yen
Original Assignee
Vertex Pharma
Crispr Therapeutics Ag
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Vertex Pharma, Crispr Therapeutics Ag filed Critical Vertex Pharma
Publication of JOP20210276A1 publication Critical patent/JOP20210276A1/ar

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/04—Centrally acting analgesics, e.g. opioids
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
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    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
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    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
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    • C12N9/14—Hydrolases (3)
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
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    • C12N2320/00—Applications; Uses
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2800/90—Vectors containing a transposable element

Abstract

يتم في هذا الطلب الكشف عن أنظمة تحرير الجين عالية الكفاءة لتحرير جين قناة صوديوم يتم التحكم فيها بالجهد، مثل الوحدة الفرعية 9 ألفا لقناة صوديوم يتم التحكم فيها بالجهد (SCN9A) أو الوحدة الفرعية 10 ألفا لقناة صوديوم يتم التحكم فيها بالجهد (SCN10A)، إما في المختبر أو في الجسم الحي. تشتمل أنظمة تحرير الجين التي تم الكشف عنها في هذا الطلب على نيوكلاز داخلي لـ DNA يتم توجيهه بـ RNA و RNAs دليلية محددة. يتم أيضاً في هذا الطلب توفير استخدامات لأنظمة تحرير الجين لتعديل الجين المستهدف، وبالتالي تخفيف الألم.
JOP/2021/0276A 2019-04-12 2020-04-10 أنظمة تحرير جيني لتعديل جين scn9a أو scn10a وطرق لاستخدامها JOP20210276A1 (ar)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962833523P 2019-04-12 2019-04-12
PCT/US2020/027690 WO2020210640A1 (en) 2019-04-12 2020-04-10 Gene-editing systems for modifying a scn9a or scn10a gene and methods of use thereof

Publications (1)

Publication Number Publication Date
JOP20210276A1 true JOP20210276A1 (ar) 2023-01-30

Family

ID=70416602

Family Applications (1)

Application Number Title Priority Date Filing Date
JOP/2021/0276A JOP20210276A1 (ar) 2019-04-12 2020-04-10 أنظمة تحرير جيني لتعديل جين scn9a أو scn10a وطرق لاستخدامها

Country Status (15)

Country Link
US (1) US20200354741A1 (ar)
EP (1) EP3953460A1 (ar)
JP (1) JP2022526670A (ar)
KR (1) KR20210151110A (ar)
CN (1) CN113994009A (ar)
AU (1) AU2020272031A1 (ar)
BR (1) BR112021020406A2 (ar)
CA (1) CA3136700A1 (ar)
CO (1) CO2021014015A2 (ar)
EA (1) EA202192806A1 (ar)
IL (1) IL287149A (ar)
JO (1) JOP20210276A1 (ar)
MX (1) MX2021012493A (ar)
SG (1) SG11202111270SA (ar)
WO (1) WO2020210640A1 (ar)

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11459587B2 (en) 2016-07-06 2022-10-04 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of pain related disorders
AU2017292169B2 (en) 2016-07-06 2021-12-23 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of pain related disorders
US11898179B2 (en) * 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing

Also Published As

Publication number Publication date
CO2021014015A2 (es) 2021-10-29
WO2020210640A1 (en) 2020-10-15
BR112021020406A2 (pt) 2021-12-07
SG11202111270SA (en) 2021-11-29
MX2021012493A (es) 2022-01-06
EA202192806A1 (ru) 2022-02-03
US20200354741A1 (en) 2020-11-12
CN113994009A (zh) 2022-01-28
IL287149A (en) 2021-12-01
AU2020272031A1 (en) 2021-11-18
CA3136700A1 (en) 2020-10-15
JP2022526670A (ja) 2022-05-25
KR20210151110A (ko) 2021-12-13
EP3953460A1 (en) 2022-02-16

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